PTC to Acquire Marathon’s Recently Approved Duchenne Muscular Dystrophy Drug

Amid a drug pricing controversy, PTC Therapeutics has agreed to buy Marathon Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug, Emflaza. DMD is a rare genetic disorder that primarily affects young boys, and Emflaza is the first treatment to be approved for the condition, regardless of the patient’s genetic mutation, in the US.

While US DMD patients were previously able to import a generic version of the drug from other countries for around $1,000, Emflaza’s US Food and Drug Administration (FDA) approval means patients could now pay as much as $89,000 per year for the drug. Public criticism over the price tag for Emflaza forced Marathon to put the brakes on the drug’s launch.

“Obviously we’re re-examining the current price of Emflaza … We believe a change needs to be had, but … it’s really too premature to speculate on the price level,” commented Dr. Stuart Peltz, CEO of PTC Therapeutics in yesterday’s conference call. The biopharmaceutical company is focused on developing drugs for rare diseases and other underserved patient communities.

PTC plans to pay Marathon $75 million in cash and $65 million in stock for commercialization rights for Emflaza. The company is also awaiting regulatory approval of its own DMD drug candidate, Translarna.

“With our nearly 20-year commitment to the Duchenne community, it is deeply meaningful for us to bring this critical therapy to US patients,” said Peltz. “We believe Emflaza is a disease-modifying therapy that has been shown to slow disease progression. In keeping with PTC’s mission, we are excited to work with the community to raise the standard of care for DMD patients.”

Emflaza is a steroid drug designed to reduce inflammation and the immune response in all patients with DMD. The only other approved treatment for the disorder is Sarepta Therapeutics’ Exondys 51, which is only indicated for DMD patients with a specific gene mutation.

“Our goal has always been to ensure Emflaza is studied, understood and available to any Duchenne patient who needs it, and we determined that this transaction is the best path for ensuring that will happen,” said Jeff Aronin, president and CEO, Marathon Pharmaceuticals. “Now that we have achieved FDA approval of Emflaza, the focus can turn to ensuring patients have access to this important therapy. PTC is well known by the Duchenne community and is ideally positioned to achieve this shared goal.”